While COVID-19 dominated the clinical data news during the month of April, with 45% due to trial delays, suspensions and terminations, and another 12% focused on therapeutic and vaccine development targeting the deadly infection, a number of companies still posted positive phase III data for other indications and are preparing for regulatory filings and commercialization.
Stamford, Conn.-based Cara Therapeutics Inc., Allschwil, Switzerland-based Idorsia Ltd., New York-based Axsome Therapeutics Inc. and Morris Plains, N.J.-based Immunomedics Inc. are among those that hit primary endpoints in pivotal studies.
Cara achieved the primary endpoint in its phase III Kalm-2 study of Korsuva (CR-845/difelikefalin), a kappa opioid receptor agonist, for injection in moderate to severe chronic disease-associated pruritis, and is set for regulatory filings for the U.S. and Europe in the second half of this year.
Idorsia’s top-line data from the first of two pivotal phase III studies showed daridorexant, a dual orexin receptor antagonist, significantly improved sleep onset, sleep maintenance and improved subjective total sleep in 930 insomnia patients. Analysts expect regulatory submissions by the end of the year.
Axsome is preparing an NDA filing in the fourth quarter for AXS-07 (meloxicam, rizatriptan) to treat acute migraine, after its Intercept phase III trial hit both co-primary endpoints of freedom from migraine pain and freedom from the most bothersome symptoms compared to placebo.
And Immunomedics already gained FDA approval of sacituzumab govitecan, branded Trodelvy, a few weeks after it stopped its phase III Ascent trial early due to compelling efficacy in triple-negative breast cancer. The approval came ahead of its June 2 PDUFA date under an accelerated pathway.
A handful of companies also reported negative news during the month of April:
Cambridge, Mass.-based Blueprint Medicines Corp.’s Ayvakit (avapritinib) failed to meet the primary endpoint in a phase III trial of improving progression-free survival vs. regorafenib in patients with locally advanced unresectable or metastatic gastrointestinal stromal tumor (GIST). The company is discontinuing development in non-PDGFRa (platelet derived growth factor receptor alpha) exon 18-mutant GIST indications.
Another failure occurred with Ann Arbor, Mich.-based Millendo Therapeutic’s Inc.’s pivotal study of livoletide for Prader-Willi syndrome (PWS), which failed to show a statistically significant improvement in hyperphagia and food-related behaviors vs. placebo. The company is discontinuing development of the ghrelin analogue in PWS.
Finally, Bridgewater, N.J.-based Menlo Therapeutics Inc. ended development of serlopitant, its NK1 receptor antagonist, following negative results for two phase III trials to treat pruritis associated with prurigo nodularis.
The 378 entries of clinical data collected by BioWorld consisted of phase I data (139), phase II data (136) and phase III data (103). A total of 171 entries were due to clinical trials affected by COVID-19, while 44 entries focused on therapeutics or vaccines for COVID-19 and its complications.
Clinical data collected in 2020, January through April, can be found at the following links:
Biopharma financings data for 2020 through May 6 can be found here: